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Find video protocols related to scientific articles indexed in Pubmed.
Forced expiratory flows' contribution to lung function interpretation in schoolchildren.
Eur. Respir. J.
PUBLISHED: 09-03-2014
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Forced expiratory flow (FEF) at low lung volumes are supposed to be better at detecting lung-function impairment in asthmatic children than a forced volume. The aim of this study was to examine whether FEF results could modify the interpretation of baseline and post-bronchodilator spirometry in asthmatic schoolchildren in whom forced expiratory volumes are within the normal range. Spirometry, with post-bronchodilator vital capacity within 10% of that of baseline in healthy and asthmatic children, was recorded prospectively. We defined abnormal baseline values expressed as z-scores <-1.645, forced expiratory volume in 1 s (FEV1) reversibility as a baseline increase >12%, FEF reversibility as an increase larger than the 2.5th percentile of post-bronchodilator changes in healthy children. Among 66 healthy and 50 asthmatic schoolchildren, only two (1.7%) children with normal vital capacity and no airways obstruction had abnormal baseline forced expiratory flow at 25-75% of forced vital capacity (FEF25-75%). After bronchodilation, among the 45 asthmatic children without FEV1 reversibility, 5 (11.1%) had an FEF25-75% increase that exceeded the reference interval. Isolated abnormal baseline values or significant post-bronchodilator changes in FEF are rare situations in asthmatic schoolchildren with good spirometry quality.
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Fetal growth restriction is worse than extreme prematurity for the developing lung.
Neonatology
PUBLISHED: 08-20-2014
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Perinatal lung growth is highly vulnerable to inflammation and intrauterine growth restriction (IUGR), two major risk factors for chronic lung disease (CLD) in preterm neonates. However, the balance between extremely low gestational age (ELGA) and IUGR in very preterm infants as risk factors for CLD and co-morbidities remains poorly explored.
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Central venous thrombosis and thrombophilia in cystic fibrosis: A prospective study.
J. Cyst. Fibros.
PUBLISHED: 08-05-2014
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Catheter venous thrombosis may result in life-threatening embolic complications. Recently, a thrombophilic tendency was described in cystic fibrosis (CF), the significance of which remains unclear. The aims of this study were to (1) document the frequency of catheter venous thrombosis detected by colour-Doppler-ultrasound (Doppler-US), (2) assess genetic and acquired thrombophilia risk factors for catheter venous thrombosis and hypercoagulability status and (3) provide recommendations on laboratory screening when considering insertion of a totally implantable vascular access device (TIVAD) in CF patients.
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Juvenile myelomonocytic leukaemia and Noonan syndrome.
J. Med. Genet.
PUBLISHED: 08-05-2014
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Infants with Noonan syndrome (NS) are predisposed to developing juvenile myelomonocytic leukaemia (JMML) or JMML-like myeloproliferative disorders (MPD). Whereas sporadic JMML is known to be aggressive, JMML occurring in patients with NS is often considered as benign and transitory. However, little information is available regarding the occurrence and characteristics of JMML in NS.
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Identification of women at high risk for severe perineal lacerations.
Eur. J. Obstet. Gynecol. Reprod. Biol.
PUBLISHED: 07-06-2014
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To evaluate the frequency and to identify the risk factors of severe perineal lacerations and the subgroup of women exposed to the highest risk for these complications.
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VITamin D supplementation in renAL transplant recipients (VITALE): a prospective, multicentre, double-blind, randomized trial of vitamin D estimating the benefit and safety of vitamin D3 treatment at a dose of 100,000 UI compared with a dose of 12,000 UI in renal transp
Trials
PUBLISHED: 06-03-2014
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In addition to their effects on bone health, high doses of cholecalciferol may have beneficial non-classic effects including the reduction of incidence of type 2 diabetes mellitus, cardiovascular disease, and cancer. These pleiotropic effects have been documented in observational and experimental studies or in small intervention trials. Vitamin D insufficiency is a frequent finding in renal transplant recipients (RTRs), and this population is at risk of the previously cited complications.
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Aspergillus in the lower respiratory tract of immunocompetent critically ill patients.
J. Infect.
PUBLISHED: 04-05-2014
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To shed light on the meaning of Aspergillus-positive lower-respiratory-tract samples in non immunocompromized critically ill patients.
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Graves' disease in children.
Best Pract. Res. Clin. Endocrinol. Metab.
PUBLISHED: 03-18-2014
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Graves' disease is an autoimmune disorder resulting from thyrotropin receptor stimulation by autoantibodies. It may occur at any age during childhood, but its frequency increases with age, peaking during adolescence. Symptoms and signs are often recognizable and proportional to the increase in serum free thyroid hormone levels. Antithyroid drug treatment with methimazole (or carbimazole) is recommended for initial treatment, but relapse rates are high, with remission achieved in only 30% of children after a first course of treatment for about two years. More prolonged medical treatment may increase the remission rate to up to 50%. Alternative treatments, such as radioactive iodine or thyroidectomy, are considered in cases of relapse, lack of compliance or antithyroid drug toxicity. Relapse risk decreases with increasing duration of the first course of antithyroid drug treatment. The identification of other predictive factors, such as severe biochemical hyperthyroidism at diagnosis, young age and the absence of other autoimmune conditions, has made it possible to stratify patients according to the risk of relapse, leading to improvements in patient management, by facilitating the identification of patients requiring long-term antithyroid drug treatment or early alternative therapy.
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Describing ICU data with tables.
Intensive Care Med
PUBLISHED: 02-10-2014
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The purpose of a scientific paper is to communicate results and within the paper this applies especially to the presentation of data. It is the universal practice in medical journals to present statistical results using tables. Good tables are an integral part of the manuscript. To help researchers communicate their results, we present practical guidance for reporting statistical results using tables.
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Comparison of two data collection processes in clinical studies: electronic and paper case report forms.
BMC Med Res Methodol
PUBLISHED: 01-17-2014
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Electronic Case Report Forms (eCRFs) are increasingly chosen by investigators and sponsors of clinical research instead of the traditional pen-and-paper data collection (pCRFs). Previous studies suggested that eCRFs avoided mistakes, shortened the duration of clinical studies and reduced data collection costs.
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Is melatonin ready to be used in preterm infants as a neuroprotectant?
Dev Med Child Neurol
PUBLISHED: 01-06-2014
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The prevention of neurological disabilities following preterm birth remains a major public health challenge and efforts are still needed to test the neuroprotective properties of candidate molecules. Melatonin serves as a neuroprotectant in adult models of cerebral ischemia through its potent antioxidant and anti-inflammatory effects. An increasing number of preclinical studies have consistently demonstrated that melatonin protects the damaged developing brain by preventing abnormal myelination and an inflammatory glial reaction, a major cause of white matter injury. The main questions asked in this review are whether preclinical data on the neuroprotective properties of melatonin are sufficient to translate this concept into the clinical setting, and whether melatonin can reduce white matter damage in preterm infants. This review provides support for our view that melatonin is now ready to be tested in human preterm neonates, and discusses ongoing and planned clinical trials.
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POPI (Pediatrics: Omission of Prescriptions and Inappropriate prescriptions): development of a tool to identify inappropriate prescribing.
PLoS ONE
PUBLISHED: 01-01-2014
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Rational prescribing for children is an issue for all countries and has been inadequately studied. Inappropriate prescriptions, including drug omissions, are one of the main causes of medication errors in this population. Our aim is to develop a screening tool to identify omissions and inappropriate prescriptions in pediatrics based on French and international guidelines.
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Hemodialysis without systemic anticoagulation: a prospective randomized trial to evaluate 3 strategies in patients at risk of bleeding.
PLoS ONE
PUBLISHED: 01-01-2014
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In this clinical trial, we aimed to compare three means of performing chronic hemodialysis in patients with contra-indication to systemic heparinization.
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Impact of glucose-6-phosphate dehydrogenase deficiency on sickle cell anaemia expression in infancy and early childhood: a prospective study.
Br. J. Haematol.
PUBLISHED: 05-23-2013
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In patients with sickle cell anaemia (SCA), concomitant glucose-6-phosphate dehydrogenase (G6PD) deficiency is usually described as having no effect and only occasionally as increasing severity. We analysed sequential clinical and biological data for the first 42 months of life in SCA patients diagnosed by neonatal screening, including 27 G6PD-deficient patients, who were matched on sex, age and parents geographic origin to 81 randomly selected patients with normal G6PD activity. In the G6PD-deficient group, steady-state haemoglobin was lower (-6·2 g/l, 95% confidence interval (CI), [-10·1; -2·3]) and reticulocyte count higher (247 × 10(9) /l, 95%CI, [97; 397]). The acute anaemic event rate was 3 times higher in the G6PD-deficient group (P < 10(-3) ). A higher proportion of G6PD-deficient patients required blood transfusion (20/27 [74%] vs. 37/81 [46%], P < 10(-3) ), for acute anaemic events, and also vaso-occlusive and infectious events. No significant between-group differences were found regarding the rates of vaso-occlusive, infectious, or cerebrovascular events. G6PD deficiency in babies with SCA worsens anaemia and increases blood transfusion requirements in the first years of life. These effects decrease after 2 years of age, presumably as the decline in fetal haemoglobin levels leads to increased sickle cell haemolysis and younger red blood cells with higher G6PD activity.
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The effect of atropine on rhythm and conduction disturbances during 322 critical care intubations.
Pediatr Crit Care Med
PUBLISHED: 05-22-2013
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Our objectives were to describe the prevalence of arrhythmia and conduction abnormalities before critical care intubation and to test the hypothesis that atropine had no effect on their prevalence during intubation.
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Adjunctive steroid in HIV-negative patients with severe Pneumocystis pneumonia.
Respir. Res.
PUBLISHED: 05-03-2013
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High-dose steroid therapy has been proven effective in AIDS-related Pneumocystis pneumonia (PCP) but not in non-AIDS-related cases. We evaluated the effects on survival of steroids in HIV-negative patients with PCP.
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Association between childhood migraine and history of infantile colic.
JAMA
PUBLISHED: 04-18-2013
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Infantile colic is a common cause of inconsolable crying during the first months of life and has been thought to be a pain syndrome. Migraine is a common cause of headache pain in childhood. Whether there is an association between these 2 types of pain in unknown.
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Quality indicator development and implementation in maternity units.
Best Pract Res Clin Obstet Gynaecol
PUBLISHED: 04-02-2013
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Measuring the quality of inpatient obstetrical care has generated considerable interest in recent years. Numerous quality measures have been proposed by national and international programmes and by obstetrics societies; however, no agreement has been reached on which measures should be used. Differences in opinions across healthcare professionals complicate the development of a standardised set of quality indicators. The use of structured methods, particularly consensus methods such as Delphi techniques, can help to choose indicators according to quality goals. Once relevant indicators are identified, maternity units should consider using a dashboard to plan and improve their services. Statistical process control is a statistical method designed to monitor and control processes. This method seems particularly promising for monitoring quality indicators in maternity units. Among statistical process control techniques, cumulative sum charts that monitor pre-selected quality indicators can be easily designed for obstetrics and gynaecology units. Cumulative sum charts provide clinicians with a picture of current practices, and rapidly detect unwanted changes in quality indicator rates.
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Recruitment in pediatric clinical research was influenced by study characteristics and pediatricians perceptions: a multicenter survey.
J Clin Epidemiol
PUBLISHED: 03-28-2013
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The aim of this survey was to quantify refusal rates and identify factors of refusal pertaining to studies and recruiting pediatricians in the research recruitment process.
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Randomized controlled trials of antibiotics for neonatal infections: a systematic review.
Br J Clin Pharmacol
PUBLISHED: 03-04-2013
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Antibiotics are a key resource for the management of infectious diseases in neonatology and their evaluation is particularly challenging. We reviewed medical literature to assess the characteristics and quality of randomized controlled trials on antibiotics in neonatal infections.
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Quality indicators for continuous monitoring to improve maternal and infant health in maternity departments: a modified Delphi survey of an international multidisciplinary panel.
PLoS ONE
PUBLISHED: 03-01-2013
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Measuring the quality of inpatient obstetrical care using quality indicators is becoming increasingly important for both patients and healthcare providers. However, there is no consensus about which measures are optimal. We describe a modified Delphi method to identify a set of indicators for continuously monitoring the quality of maternity care by healthcare professionals.
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Experimental designs for small randomised clinical trials: an algorithm for choice.
Orphanet J Rare Dis
PUBLISHED: 02-21-2013
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Small clinical trials are necessary when there are difficulties in recruiting enough patients for conventional frequentist statistical analyses to provide an appropriate answer. These trials are often necessary for the study of rare diseases as well as specific study populations e.g. children. It has been estimated that there are between 6,000 and 8,000 rare diseases that cover a broad range of diseases and patients. In the European Union these diseases affect up to 30 million people, with about 50% of those affected being children. Therapies for treating these rare diseases need their efficacy and safety evaluated but due to the small number of potential trial participants, a standard randomised controlled trial is often not feasible. There are a number of alternative trial designs to the usual parallel group design, each of which offers specific advantages, but they also have specific limitations. Thus the choice of the most appropriate design is not simple.
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Triage of children with headache at the ED: a guideline implementation study.
Am J Emerg Med
PUBLISHED: 02-04-2013
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We present a multicenter validation of a modified Manchester Triage System (MTS) flowchart for pediatric patients who present with headache to the emergency department. A prospective observational study was conducted across 5 European pediatric emergency departments. The standard MTS headache flowchart and a modified MTS headache flowchart were tested in the participating centers, and results were compared with triage categories identified by either the physician at the end of the clinical examination or the reference classification matrix (RCM). Fifty-three patients were enrolled in the preimplementation phase and 112 in the postimplementation phase. When compared with physicians triage and RCM, the modified MTS flowchart demonstrated good sensitivity (79% and 70%, respectively), specificity (77% and 76%, respectively), and a high positive likelihood ratio (9.14 and 16.75, respectively) for the identification of low-risk children. Conclusions: Our modified headache flowchart is safe and reliable in pediatric emergency settings, especially for lower classes of urgency.
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Assessment of upper limb function in young Friedreich ataxia patients compared to control subjects using a new three-dimensional kinematic protocol.
Clin Biomech (Bristol, Avon)
PUBLISHED: 01-25-2013
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The assessment of Friedreich ataxia effects on upper limb function in clinical follow-up remains a challenging issue. To complete the usual clinical scales, an upper limb kinematic protocol adapted to Friedreich ataxia children and young adults has been developed and applied to both patients and control subjects.
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Atropine for critical care intubation in a cohort of 264 children and reduced mortality unrelated to effects on bradycardia.
PLoS ONE
PUBLISHED: 01-21-2013
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Atropine has is currently recommended to facilitate haemodynamic stability during critical care intubation. Our objective was to determine whether atropine use at induction influences ICU mortality.
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Randomized controlled trial of parent therapeutic education on antibiotics to improve parent satisfaction and attitudes in a pediatric emergency department.
PLoS ONE
PUBLISHED: 01-01-2013
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To evaluate therapeutic education delivered in a pediatric emergency department to improve parents satisfaction and attitudes about judicious antibiotic use.
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How to improve the implementation of academic clinical pediatric trials involving drug therapy? A qualitative study of multiple stakeholders.
PLoS ONE
PUBLISHED: 01-01-2013
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The need for encouraging pediatric drug research is widely recognized. However, hospital-based clinical trials of drug treatments are extremely time-consuming, and delays in trial implementation are common. The objective of this qualitative study was to collect information on the perceptions and experience of health professionals involved in hospital-based pediatric drug trials.
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Positive impact of long-term antithyroid drug treatment on the outcome of children with Graves disease: national long-term cohort study.
J. Clin. Endocrinol. Metab.
PUBLISHED: 10-26-2011
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Drug-based therapy is usually the initial treatment for Graves disease (GD) hyperthyroidism in children, but there is some debate about treatment duration.
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Serum concentrations of insulin-like growth factor (IGF)-1 and IGF binding protein-3 (IGFBP-3), IGF-1/IGFBP-3 ratio, and markers of bone turnover: reference values for French children and adolescents and z-score comparability with other references.
Clin. Chem.
PUBLISHED: 08-24-2011
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A reference model for converting serum growth factor and bone metabolism markers into an SD score (SDS) is required for clinical practice. We aimed to establish reference values of serum insulin-like growth factor-1 (IGF-1) and IGF binding protein 3 (IGFBP-3) concentrations and bone metabolism markers in French children, to generate a model for converting values into SDS for age, sex, and pubertal stage.
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Mycophenolate mofetil for steroid-dependent nephrotic syndrome: a phase II Bayesian trial.
Pediatr. Nephrol.
PUBLISHED: 07-13-2011
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Mycophenolate mofetil (MMF) has emerged as a new therapeutic option in steroid-dependent nephrotic syndrome (SDNS). We conducted a phase II Bayesian trial of MMF in children with SDNS. Phase II trials, usually single-arm studies, investigate the effect of new treatments. Standard Flemings procedure relies on observed results (relapse rate during the trial), whereas Bayesian approach combines observed results with prior information (expected relapse rate according to prior studies and clinical experience). All patients were required to have received prior alkylating-agent treatment. Sixty-seven percent of them had also received levamisole. Patients received MMF (1,200 mg/m(2)/day) and prednisone according to a defined schedule [reduction of alternate-day (e.o.d) dose to 50% of pre-MMF dose at 3 months, 25% at 6 months]. Twenty-four children (median age 6.0 years, 2.8-14.4) entered the study and 23 completed it. Bayesian analysis showed that adding four patients would not change significance of results, allowing stopping inclusions. Four patients relapsed during the first 6 months (estimated probability 17.6%, 95% credibility interval: 5.4-35.0%) and two at months 8 and 11.5. In the 19 patients free of relapse during the first 6 months, median (Q1-Q3) prednisone maintenance dose decreased from 25 (10-44) to 9 (7.5-11.2) mg/m(2) e.o.d (p < 0.001) and cumulative dose from 459 (382-689) to 264 (196-306) mg/m(2)/month (p < 0.001) before and on MMF respectively. Pre-MMF patient characteristics and MMF pharmacokinetics did not differ between patients with or without relapse. MMF reduces relapse rate and steroid dose in children with SDNS and should be proposed before cyclosporine and cyclophosphamide.
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Normal ranges and genetic variants of antithrombin, protein C and protein S in the general Chinese population. Results of the Chinese Hemostasis Investigation on Natural Anticoagulants Study I Group.
Haematologica
PUBLISHED: 04-12-2011
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Inherited deficiency of antithrombin, protein C and protein S, three important, naturally occurring coagulation inhibitors, might play a major role in the occurrence of venous thromboembolism in Chinese. The establishment of age- and gender-related normal ranges of these inhibitors is crucial for an accurate diagnosis of these deficiencies.
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Using and reporting the Delphi method for selecting healthcare quality indicators: a systematic review.
PLoS ONE
PUBLISHED: 03-22-2011
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Delphi technique is a structured process commonly used to developed healthcare quality indicators, but there is a little recommendation for researchers who wish to use it. This study aimed 1) to describe reporting of the Delphi method to develop quality indicators, 2) to discuss specific methodological skills for quality indicators selection 3) to give guidance about this practice.
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Preterm delivery disrupts the developmental program of the cerebellum.
PLoS ONE
PUBLISHED: 03-15-2011
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A rapid growth in human cerebellar development occurs in the third trimester, which is impeded by preterm delivery. The goal of this study was to characterize the impact of preterm delivery on the developmental program of the human cerebellum. Still born infants, which meant that all development up to that age had taken place in-utero, were age paired with preterm delivery infants, who had survived in an ex-utero environment, which meant that their development had also taken place outside the uterus. The two groups were assessed on quantitative measures that included molecular markers of granule neuron, purkinje neuron and bergmann glia differentiation, as well as the expression of the sonic hedgehog signaling pathway, that is important for cerebellar growth. We report that premature birth and development in an ex-utero environment leads to a significant decrease in the thickness and an increase in the packing density of the cells within the external granular layer and the inner granular layer well, as a reduction in the density of bergmann glial fibres. In addition, this also leads to a reduced expression of sonic hedgehog in the purkinje layer. We conclude that the developmental program of the cerebellum is specifically modified by events that follow preterm delivery.
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Cyclophosphamide in steroid-dependent nephrotic syndrome.
Pediatr. Nephrol.
PUBLISHED: 02-07-2011
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In order to determine the long-term effects of cyclophosphamide (CPO) and to identify parameters associated with sustained remission, we retrospectively studied the data from 90 patients with steroid-dependent nephrotic syndrome (SDNS) who received a single course of oral cyclophosphamide (2 mg/kg/day for 10 to 12 weeks). The median follow-up period after CPO was 5.5 years (interquartile range 3.2-8.5). Sustained remission reached the cumulative rate of 57% at 1 year, 42% at 2 years, and 31% at 5 years. For the patients who relapsed, the median threshold dose of prednisone between CPO initiation and first relapse has significantly decreased (22.1 mg/kg/day versus 4.9 mg/kg/day, p?
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Iodine deficiency in northern Paris area: impact on fetal thyroid mensuration.
PLoS ONE
PUBLISHED: 01-10-2011
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Iodine is essential for normal fetal and neonatal development. We studied the prevalence and impact on fetal thyroid development of iodine deficiency in pregnant women in the northern part of the Paris conurbation.
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Comprehensive description of CFTR genotypes and ultrasound patterns in 694 cases of fetal bowel anomalies: a revised strategy.
Hum. Genet.
PUBLISHED: 08-02-2010
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Fetal bowel anomalies may reveal cystic fibrosis (CF) and the search for CF transmembrane conductance regulator (CFTR) gene mutations is part of the diagnostic investigations in such pregnancies, according to European recommendations. We report on our 18-year experience to document comprehensive CFTR genotypes and correlations with ultrasound patterns in a series of 694 cases of fetal bowel anomalies. CFTR gene analysis was performed in a multistep process, including search for frequent mutations in the parents and subsequent in-depth search for rare mutations, depending on the context. Ultrasound patterns were correlated with the genotypes. Cases were distinguished according to whether they had been referred directly to our laboratory or after an initial testing in another laboratory. A total of 30 CF fetuses and 8 cases compatible with CFTR-related disorders were identified. CFTR rearrangements were found in 5/30 CF fetuses. 21.2% of fetuses carrying a frequent mutation had a second rare mutation, indicative of CF. The frequency of CF among fetuses with no frequent mutation was 0.43%. Correlation with ultrasound patterns revealed a significant frequency of multiple bowel anomalies in CF fetuses. The results emphasize the need to search for rearrangements in the diagnosis strategy of fetal bowel anomalies. The diagnostic value of ultrasound patterns combining hyperechogenic bowel, loop dilatation and/or non-visualized gallbladder reveals a need to revise current strategies and to offer extensive CFTR gene testing when the triad is diagnosed, even when no frequent mutation is found in the first-step analysis.
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Addition of cyclophosphamide to steroids provides no benefit compared with steroids alone in treating adult patients with severe Henoch Schönlein Purpura.
Kidney Int.
PUBLISHED: 05-26-2010
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Henoch Schönlein Purpura (HSP) is a common disease in children, usually associated with a good prognosis. In adults there are no prospective studies concerning its prognosis or treatment, especially in cases of severe visceral involvement. Here we compared steroid therapy without or with cyclophosphamide co-treatment in adults with severe HSP in a 12-month, multi-center, prospective, open-label trial that treated 54 adults with biopsy-proven HSP including proliferative glomerulonephritis and severe visceral manifestations. All received steroids; however, 25 were randomized to also receive cyclophosphamide. The primary endpoint that occurred in three patients in each group was complete disease remission defined as zero on the Birmingham Vasculitis Activity Score with no persistent or new clinical and/or biological vasculitis at 6 months. No patient had active visceral involvement. The secondary endpoints were renal outcome, deaths, and adverse events at 12 months. Renal function, proteinuria, safety data, incidence of diabetes, and severe infections were similar between the two groups. At the last follow-up, renal function remained stable. The small population size of our study does not permit definitive conclusions; however, we suggest that treatment of adults with severe HSP by adding cyclophosphamide provides no benefit compared with steroids alone.
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Are cytochrome P450 CYP2C8 and CYP2C9 polymorphisms associated with ibuprofen response in very preterm infants?
PLoS ONE
PUBLISHED: 04-23-2010
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Patent ductus arteriosus (PDA) in extremely preterm infants remains a challenging condition with conflicting treatment strategies. Ibuprofen is currently used to treat PDA with ductal closure failure rate up to 40%. We test the hypothesis that cytochrome P450 CYP2C8/2C9 polymorphisms may predict ibuprofen response.
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Fewer infants than older patients in paediatric randomised controlled trials.
Eur. J. Epidemiol.
PUBLISHED: 03-09-2010
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To determine whether the youngest age groups are less likely to be included in paediatric randomised controlled trials (PRCTs) than older children, we conducted a PubMed search using the keyword "randomised controlled trial" and the limit "all child: 0-18 years". We retrieved 417 articles published in 2006 in 34 leading journals classified as general medical journals, paediatric medical journals, or specialist medical journals. We arbitrarily selected 144 articles, at random. For each study, we evaluated population age characteristics (central tendency, range, and dispersion), study design, sample size and topic. Of the 144 studies, only 82 were first reports of paediatric randomised controlled trials (PRCTs). Among the other studies, many were done in adults. Of the 82 PRCTs, only 11% included newborns and 26% infants; 59% included children and 39% adolescents. Using the same search strategy to retrieve PRCTs in the same journals in the last 4 months of 2009 retrieved 66 PRCTs, of which 17% included newborns, 24% infants, 61% children and 55% adolescents. The three health conditions most often reported were respiratory diseases, infectious diseases, and mental and behavioural disorders. In 34 leading journals, PRCTs were significantly less likely to include newborns and infants than older paediatric patients. Given the huge impact of PRCTs on paediatric health, additional efforts are needed to promote studies in newborns and infants, as well as studies of the impact of recent European and American regulations designed to encourage paediatric drug trials.
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Randomized study of the effect of topical anesthesia on tuberculin skin test reaction size in children.
Pediatr. Infect. Dis. J.
PUBLISHED: 02-06-2010
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Tuberculin skin test (TST) application in children can be eased by topical anesthesia, but no study has determined whether lidocaine-prilocaine mixture application modifies TST skin reactions. We compared TST performed with and without topical anesthesia in 46 children (range, 0.4-15.9 years), and found that topical lidocaine-prilocaine did not affect the TST size reaction. Topical lidocaine-prilocaine can be used for TST.
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Genotype-phenotype correlations in fetuses and neonates with autosomal recessive polycystic kidney disease.
Kidney Int.
PUBLISHED: 11-25-2009
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The prognosis of autosomal recessive polycystic kidney disease is known to correlate with genotype. The presence of two truncating mutations in the PKHD1 gene encoding the fibrocystin protein is associated with neonatal death while patients who survive have at least one missense mutation. To determine relationships between genotype and renal and hepatic abnormalities we correlated the severity of renal and hepatic histological lesions to the type of PKHD1 mutations in 54 fetuses (medical pregnancy termination) and 20 neonates who died shortly after birth. Within this cohort, 55.5% of the mutations truncated fibrocystin. The severity of cortical collecting duct dilatations, cortical tubule and glomerular lesions, and renal cortical and hepatic portal fibrosis increased with gestational age. Severe genotypes, defined by two truncating mutations, were more frequent in patients of less than 30 weeks gestation compared to older fetuses and neonates. When adjusted to gestational age, the extension of collecting duct dilatation into the cortex and cortical tubule lesions, but not portal fibrosis, was more prevalent in patients with severe than in those with a non-severe genotype. Our results show the presence of two truncating mutations of the PKHD1 gene is associated with the most severe renal forms of prenatally detected autosomal recessive polycystic kidney disease. Their absence, however, does not guarantee survival to the neonatal period.
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Proficiency program for real-time PCR diagnosis of Bordetella pertussis infections in French hospital laboratories and at the French National Reference Center for Whooping Cough and other Bordetelloses.
J. Clin. Microbiol.
PUBLISHED: 08-19-2009
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With the support of a ministerial program for innovative and expensive technologies, dedicated to the economic evaluation of laboratory diagnosis of pertussis by real-time PCR, external quality assessment for real-time IS481 PCR was carried out. Coordinated by the National Centre of Reference of Pertussis and other Bordetelloses (NCR), this study aimed to harmonize and to assess the performances of eight participating microbiology hospital laboratories throughout the French territory. Between January 2006 and February 2007, 10 proficiency panels were sent by the NCR (ascending proficiency program), representing a total of 49 samples and including eight panels to analyze and evaluate the global sensitivity and specificity of real-time PCR, one to assess the limit of detection, and one to evaluate nucleic acid extraction methods. As part of the descending proficiency program, extracted DNA from clinical samples was sent by the eight participating laboratories in different panels and analyzed by the NCR. In the ascending proficiency analysis, the sensitivity and specificity of the real-time PCR methods were 92.2% and 94.3%, respectively. The limit of detection of the different methods ranged between 0.1 and 1 fg/microl (0.2 to 2 CFU/microl). The nucleic acid extraction methods showed similar performances. During the descending proficiency analysis, performed with 126 samples, the result of the NCR for 15 samples (11.9%) was discordant with the result obtained by the source laboratory. Despite several initial differences, harmonization was easy and performances were homogeneous. However, the risk of false-positive results remains quite high, and we strongly recommend establishment of uniform quality control procedures performed regularly.
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Cost effectiveness of sirolimus-eluting stents compared with bare metal stents in acute myocardial infarction: insights from the TYPHOON trial.
Appl Health Econ Health Policy
PUBLISHED: 06-30-2009
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Drug-eluting stents have been shown to reduce the rate of repeat revascularization after percutaneous coronary intervention for acute myocardial infarction (AMI) as compared with bare metal stents (BMS). A few studies have reported the cost effectiveness of sirolimus-eluting stents (SES) in several countries, but none in the particular setting of AMI in France.
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Growth in boys with idiopathic nephrotic syndrome on long-term cyclosporin and steroid treatment.
Pediatr. Nephrol.
PUBLISHED: 06-15-2009
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Although steroid-free remission can usually be achieved with cyclosporin A (CsA) in patients with steroid-dependent nephrotic syndrome (SDNS), some CsA-treated patients require long-term steroid therapy. Data on growth in these patients are scarce. Sixty-four boys with SDNS receiving long-term CsA and steroid therapy were retrospectively analyzed. During the 10-year follow-up period, height standard deviation score (HSDS) remained in the normal range in 47 patients but was below -2 SD in 17 patients. The occurrence of growth retardation was influenced by height at diagnosis and the number of relapses. Thirty patients were followed for at least 3 years before and after age 12. The decrease in HSDS per year of disease in patients older than 12 years was twice that observed in children younger than 12. However, adult height was < or = -2 SD in only two of the 14 patients reaching adult height, reflecting potential catch-up growth during late puberty. Careful monitoring of growth is recommended, given than up to 25% of patients experienced severe growth retardation during the course of their disease.
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Lack of functional benefit with glutamine versus placebo in Duchenne muscular dystrophy: a randomized crossover trial.
PLoS ONE
PUBLISHED: 04-08-2009
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Oral glutamine decreases whole body protein breakdown in Duchenne muscular dystrophy (DMD). We evaluated the functional benefit of 4 months oral glutamine in DMD.
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Effect of transfusion therapy on cerebral vasculopathy in children with sickle-cell anemia.
Haematologica
PUBLISHED: 02-13-2009
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This retrospective study assessed the long-term effect of transfusional exchange therapy on MRA/MRI abnormalities in 24 homozygous sickle-cell anemia (HbSS) children presenting with abnormal brain MRA. The median time elapsed from baseline to last available MRA was 29 months. Follow-up MRAs showed improvement, stabilization or worsening of cerebrovascular lesions in 11, 6 and 7 patients respectively. Complete normalization of MRA was observed in 6 patients within a mean time of 1.4 years, but stenosis recurred at the same location in the 4 patients in whom transfusion therapy was discontinued. Baseline severe stenosis/occlusion of large cerebral arteries and occurrence of moyamoya syndrome were significantly associated with an absence of improvement of the cerebral vasculopathy. These data emphasize the heterogeneity of the course of cerebrovasculopathy in SS children receiving chronic transfusion. Further studies are needed to determine whether different therapeutic approaches have to be considered according to these different evolutive patterns in SS children.
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Crohns disease and early exposure to domestic refrigeration.
PLoS ONE
PUBLISHED: 01-29-2009
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Environmental risk factors playing a causative role in Crohns Disease (CD) remain largely unknown. Recently, it has been suggested that refrigerated food could be involved in disease development. We thus conducted a pilot case control study to explore the association of CD with the exposure to domestic refrigeration in childhood.
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Peer review of grant applications: criteria used and qualitative study of reviewer practices.
PLoS ONE
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Peer review of grant applications has been criticized as lacking reliability. Studies showing poor agreement among reviewers supported this possibility but usually focused on reviewers scores and failed to investigate reasons for disagreement. Here, our goal was to determine how reviewers rate applications, by investigating reviewer practices and grant assessment criteria.
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Effect of glutamine on glucose metabolism in children with Duchenne muscular dystrophy.
Clin Nutr
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Glutamine is a potent gluconeogenic precursor and stimulates insulin secretion. Glutamines effect on glucose metabolism in Duchenne muscular dystrophy (DMD) has never been studied. To determine plasma glucose and insulin concentrations measured during and after glutamine administration in DMD boys. We hypothesized that glutamine can modulate whole body glutamine-glucose metabolism in DMD, a genetically determined disease.
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Stature is an essential predictor of muscle strength in children.
BMC Musculoskelet Disord
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Children with growth retardation or short stature generally present with lower strength than children of the same chronological age. The aim of the study was to establish if strength was dependent on variables related to stature in a population of healthy children and to propose practical predictive models for the muscle functions tested. A secondary aim was to test for any learning effects concerning strength measured at two successive visits by children.
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Diagnosis delay in West syndrome: misdiagnosis and consequences.
Eur. J. Pediatr.
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West syndrome or infantile spasms is one of the most frequent epileptic syndromes in the first year of life. The clinical symptoms of infantile spasms are very different than any other type of seizure because of both the absence of paroxysmal motor phenomena (i.e., as in a convulsion) and the lack of significant duration of loss of consciousness (i.e., as in absence epilepsy). Infantile spasms may lead to misdiagnosis by pediatricians and other primary care providers. We assessed the missed diagnoses prior to the diagnosis of infantile spasms. We hypothesized that a delay in treatment may have consequences on neurologic outcome. We conducted a multicenter, retrospective, observational study to evaluate occurrence of misdiagnosis and its possible consequences. We performed a multivariate analysis to evaluate the risk for the outcome 2 years after the diagnosis of infantile spasms. We included 83 infants over a 5-year period. The majority of consulted physicians (301 of 362) did not suggest any specific diagnosis while the others suggested gastroesophageal reflux (7 %), constipation (7 %), or colitis (3 %). Results indicated that a poor outcome was related to a delay in diagnosis, which was observed regardless of the existence of cognitive involvement prior to the start of infantile spasms (Relative Risk: RR 12.08 [1.52-96.3]). These results highlight the importance of making an early diagnosis of infantile spasms.
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[Quality of life, asthma control, urinary cotinine and therapeutic education among asthmatic children].
Sante Publique
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Therapeutic patient education (TPE) has been standard practice in France for roughly 20 years. However, TPE has only recently been officially recognized and has rarely been assessed. The objective of this study was to assess the impact of TPE on passive smoking exposure and asthma outcomes in young patients referred to a hospital school for children with asthma. A prospective cohort study was conducted to determine the socio-economic status, environmental exposure (including parental tobacco consumption) and medical history of children starting out on a TPE course. The study measured quality of life and urinary cotinine. Asthma control was also assessed. Out of a total of 54 children (median age 8.6 years), 35 and 26 children attended three or at least four TPE sessions, respectively. Uncontrolled asthma and a history of hospitalization for exacerbation of asthma were frequent (43% and 61%, respectively). In utero passive smoke exposure was more frequent in the hospitalization group (p = 0.07). Urinary cotinine levels were similar in children exposed and not exposed to tobacco smoke (259 vs 141 nMol.L-1, p = 0.15), but only decreased in the group of exposed children. In 34 children, quality of life improved significantly between the first and the third or fourth sessions (n = 23, median increase 1.06, p = 0.1), while asthma control improved in 64% of the patients (n = 33, p = 0.01) and emergency attendances decreased (n = 34, p = 0.001). The positive effects of TPE on asthma control and quality of life were quickly visible but did not prevent children from withdrawing from the program. Urinary cotinine was not useful for detecting exposure to tobacco smoke, but may be useful as an indicator of exposure to tobacco smoke. The results indicate an improvement in quality of life and asthma control as TPE proceeded.
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Factors influencing neurological outcome of children with bacterial meningitis at the emergency department.
Eur. J. Pediatr.
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We performed a cohort study of children who survived bacterial meningitis after the neonatal period at a single pediatric center in France over a 10-year period (1995-2004) to identify predictors of death and long-term neurological deficits in children with bacterial meningitis. We performed multivariate regression to determine independent predictors of death and neurologic deficits. We identified 101 children with bacterial meningitis of which 19 died during initial hospitalization. Need for mechanical ventilation [hazard ratio (HR) 11.5, 95 % confidence interval (CI) 2.4-55.5)] and thrombocytopenia defined as a platelet count <150?×?10(9) per liter (HR 0.6, 95 % CI 0.4-0.9) at presentation were associated with death during initial hospitalization. At final assessment, 42 of the 70 survivors had no neurologic deficits identified; 20 had a single deficit, and eight had multiple deficits. A delay in initiation of antibiotics (HR 1.3, 95 % CI 1.1-1.7) and hydrocephalus on computed tomographic scan (HR 2.6, 95 % CI 1.1-6.0) were associated with having one or more long-term neurologic deficits. Identification of children at risk of death or long-term neurologic sequelae may allow therapeutic interventions to be directed to children at the highest risk.
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Non-financial conflicts of interest in academic grant evaluation: a qualitative study of multiple stakeholders in France.
PLoS ONE
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Peer review is the most widely used method for evaluating grant applications in clinical research. Criticisms of peer review include lack of equity, suspicion of biases, and conflicts of interest (CoI). CoIs raise questions of fairness, transparency, and trust in grant allocation. Few observational studies have assessed these issues. We report the results of a qualitative study on reviewers and applicants perceptions and experiences of CoIs in reviews of French academic grant applications.
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Novel modeling of reference values of cardiovascular risk factors in children aged 7 to 20 years.
Pediatrics
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Most of the cardiovascular risk factors strongly associated with obesity and overweight vary with age and gender. However, few reference values are available for healthy European children. Our objective was to establish pediatric reference ranges for waist circumference, systolic and diastolic blood pressures, fasting lipid levels (total cholesterol, high-density lipoprotein cholesterol, low-density lipoprotein cholesterol, and triglycerides), glucose, and insulin.
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Aetiology and epidemiology of fever in children presenting to the emergency department of a French paediatric tertiary care centre after international travel.
Arch. Dis. Child.
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As few data are available on the causes of fever in children returning from international travel, the authors studied children presenting to a French tertiary care centre with fever.
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Diet or medically treated gestational diabetes: is there any difference for obstetrical and neonatal complications? A French cohort study.
J Perinat Med
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Abstract Objective: The aim of the present study was to determine specific obstetrical and neonatal complications associated with diet-treated gestational diabetes (DTGD) and medically treated gestational diabetes (MTGD). Methods: This is a prospective cohort study of women followed in the Robert Debré Hospital (France, Paris) and who have given birth between 1 January, 2004, and 19 November, 2010. Clinical, biological, maternal and neonatal data were reported in the maternity database. Associations between obstetrical and neonatal complications and gestational diabetes were evaluated by estimating odd ratios (ORs) and their 95% CIs, using a logistic regression model. Results: 16,244 pregnancies were included in the study. 1515 (9.3%) women had gestational diabetes: 1108 (7.3%) had DTGD, 243 (1.7%) had MTGD. After full adjustment, MTGD was associated with an increased risk of nonscheduled cesarean (ORnonscheduled=2.3; 95% CI: 1.6-3.3; P<0.001) while DTGD was not (ORnonscheduled=1.0; 95% CI: 0.8-1.3; P<0.96). Clinical macrosomia was positively associated with DTGD (OR=2; 95% CI: 1.7-2.4; P<0.0001) or MTGD (OR=2.9; 95% CI: 2.1-3.9; P<0.0001). Conclusion: This study confirms that macrosomia is the main complication of DTGD. By contrast, DTGD was not associated with neonatal hypoglycemia and cesarean, while these complications were associated with MTGD.
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Impact of Unlabeled French Antibiotic Guidelines on Antibiotic Prescriptions for Acute Respiratory Tract Infections in Seven Pediatric Emergency Departments, 2009-2012.
Pediatr. Infect. Dis. J.
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From November 2009 to October 2012, implementation of guidelines, unlabeled by the French Agency of Health Products, changed the categories of antibiotics prescribed for acute respiratory tract infections (ARTI) in seven pediatric emergency departments. During the study, 36,413 ARTI-related antibiotic prescriptions were prescribed. Amoxicillin prescriptions rose from 30.0% to 84.7%, while amoxicillin-clavulanate and cefpodoxime prescriptions decreased to 10.2% and 2.5%, respectively.
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Adult Social and Professional Outcomes of Pediatric Renal Transplant Recipients.
Transplantation
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Little is known about the socioprofessional situation of adult-aged kidney-transplanted children. This nationwide French cohort study documented the socioprofessional outcomes of adults who underwent kidney transplantation before age 16 years between 1985 and 2002.
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